FDA Approves First Drug for Sickle Cell in 20 Years


Endari is only the second FDA-approved sickle cell disease drug.

The FDA has approved Endari (L-glutamine oral powder, Emmaus Medical), the first new drug for treating complications associated with sickle cell disease to be approved in 20 years and only the second approved overall. Endari received an orphan drug designation from the FDA.

Sickle cell disease, sometimes called sickle cell anemia, is a rare, genetic blood disorder where red blood cells are shaped like crescents or sickles. The misshaped cells tend to clump and clog blood vessels, cutting off oxygen and causing episodes of severe pain that can lead to organ damage. The condition was in the news recently because of the death of noted rapper Prodigy, who died at age 42 from complications of sickle cell disease. Most of the 100,000 people in the United States with sickle cell disease are African American or Latino.

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Clinical trials conducted by Emmaus showed that using Endari for 48 weeks reduced the frequency and length of hospital visits for sickle cell crises compared to placebo. Patients treated with Endari experienced fewer hospitalizations for sickle cell pain (median 2 compared to median 3), and fewer days in the hospital (median 6.5 days compared to median 11 days). They also had fewer occurrences of acute chest syndrome, a life-threatening complication of sickle cell disease, compared with patients who received a placebo (8.6% compared to 23.1%).

Common side effects with Endari include constipation, nausea, headache, abdominal pain, coughing, pain in the extremities, and back and chest pain.

However, L-glutamine has been on the market for many years and can be purchased as a dietary supplement without a prescription. This may affect the way health insurers cover Endari, and some may refuse to cover it.

Other treatments for sickle cell disease-treatments that work on the cause rather than the symptoms or complications-are being developed, including a gene therapy from Bluebird Bio and methods of gene editing using CRISPR technology.

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